The ability to prescribe drugs for uses beyond the officially approved indications is commonly used to good effect by healthcare providers.
However, healthcare providers are not required to limit prescriptions or recommendations to the indications approved by their country's drug regulatory body.
In fact, the standard of care for many conditions involves off-label uses, either as first-line therapy or as a subsequent line.
Regulatory approval for an indication requires a body of evidence that costs money to assemble, and as with evidence-based medicine generally, the desire for a vast, high-quality evidence base is an ideal that real-world practice can only aspire to and further approach, rather than completely match; there may not be enough resources to test every drug for every possible or logical indication to an exhaustive degree.
By some estimates, the number of clinical trials performed in children from 2002 to 2012 exceeded that in the prior 50 years.
The article recommends to pediatricians that "Off-label use is neither incorrect nor investigational if based on sound scientific evidence, expert medical judgment, or published literature" and that "Evidence, not label indication, remains the gold standard from which practitioners should draw when making therapeutic decisions for their patients."
[8] A small market and the high risk of medicolegal action, as exemplified by the Bendectin case, may explain the reluctance to develop drugs for approval.
In the United States in 2017, the government is considering allowing direct-to-consumer advertising to promote off-label drug use.
[16] The appointment of Scott Gottlieb to become head of the United States Food and Drug Administration (FDA) furthered discussion, as this person advocates to allow that sort of promotion.
[22] The FDA does not have the legal authority to regulate the practice of the medicine, and the physician may prescribe a drug off-label.
[19] Contrary to popular notion, it is legal in the United States and in many other countries to use drugs off-label, including controlled substances such as opiates.
However, in December 2012, the United States Second Circuit Court found that promotion of off-label uses by a company sales representative was considered to be protected speech per the First Amendment.
[24] In addition, the Food and Drug Administration Modernization Act of 1997 created an exception to the prohibition of off-label marketing, allowing manufacturers to provide medical practitioners with publications on off-label uses of a drug, in response to an unsolicited request.
[25] In 2004, the federal government and whistleblower David Franklin reached a $430 million settlement in Franklin v. Parke-Davis to resolve claims that Warner-Lambert engaged in off-label promotion of Neurontin in violation of the FDCA and the False Claims Act.
The labels for the two drugs were similar, but doctors prescribed Lovaza for people who had triglycerides lower than 500 mg/dL based on some clinical evidence.
According to General Medical Council guidance, the physician must be satisfied that there is sufficient evidence or experience of using the medicine to demonstrate safety and efficacy.
The FDA specifically prohibits extralabel use of a number of antibiotics, anti-inflammatory drugs and hormones in food producing animals.